【medical-news】FDA批准诺德人体生长激素用于治疗罕见遗传疾病引起的身材矮小-2010-06-21 10:17:55 AM
【medical-news】FDA批准诺德人体生长激素用于治疗罕见遗传疾病引起的身材矮小
Main Category: Endocrinology News
Article Date: 02 Jun 2007 - 0:00 PDT
Noo Nordisk today announced that Norditropin(R) (somatropin[rDNA origin] injection)receied U.S. Food and Drug Administration (FDA) approal for the treatment of short stature in children with Noonan syndrome. Noonan syndrome is defined as an autosomal dominant genetic syndrome commonly characterized by short stature, congenital heart defects, and unique facial features. The appearance of this disorder can include hypertelorism (widely-set eyes), down-slanting eyes, webbed neck, and other conditions, including congenital heart disease in half of those affected. Up to 80 percent of children with Noonan syndrome suffer from significant short stature.
"When you see a child who has Noonan syndrome, it may not always be obious to the naked eye, but the complications associated with the condition are quite serious and can affect both physical deelopment and other systems" said Martin Soeters, president of Noo Nordisk Inc. "There are few treatment options aailable to help the physical deelopment, and this approal marks an exciting adancement for children with this rare condition."
Noonan syndrome is classified as a rare condition with a population of less than 200,000. To encourage the deelopment of treatments for rare disorders -- that may not otherwise be commercially iable for deelopment -- the FDA designates drugs that treat fewer than 200,000 patients with an "orphan drug" designation. Norditropin has receied orphan drug designation for the treatment of short stature associated with Noonan syndrome.
About Noonan Syndrome
The prealence of Noonan syndrome has not been determined accurately to date, but most authors report 1 in 1,000 - 2,500 lie births, affecting males and females equally. Based on the United States population, prealence may range anywhere from 125,000 to 300,000 lie births. Howeer, fetal loss can occur in Noonan syndrome so actual incidence of the disorder may be higher than its prealence.
Clinical Features and Complications
-- Unique facial features, such as widely-spaced eyes, triangular face, low-set ears, and short-webbed neck
-- Short stature (up to 80% of indiiduals)
-- Congenital heart defects
-- Abnormal chest (shrunken sternum or concae chest)
-- Undescended testes in males (more than 50%)
-- Bleeding disorders, and easy bruising, especially Factor XI (clotting factor) deficiency (which causes Hemophilia C)
-- Feeding difficulties with babies, including poor suckling and weaning; frequent or forceful omiting may also occur
-- Common eye problems including near sightedness and a squint
-- Hearing problems caused by middle ear infections
-- Poor muscle tone in early deelopment
-- Lymphatic system problems, such as lymphedema
There is no specific pharmacologic therapy currently aailable, and treatment for Noonan syndrome focuses on its clinical features and complications.
"Noonan syndrome is a heterogeneous genetic condition in which the clinical features are quite ariable. Short stature, which can be seere, is one of the most common characteristics. Treatment with Norditropin may help children with Noonan syndrome improe one of the most concerning physical features of the condition," said Alicia Romano, M.D., Pediatric Endocrinologist, New York Medical College.
About Norditropin
Norditropin(R) (somatropin [rDNA origin] injection) is indicated for the treatment of children with short stature associated with Noonan syndrome, treatment of children with growth failure due to inadequate secretion of endogenous growth hormone and for replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD) who meet either of the following two criteria:
1) Adult Onset: Patients who hae GHD, either alone or associated with multiple hormone deficiencies (hypopituitarism), as a result of pituitary disease, hypothalamic disease, surgery, radiation therapy, or trauma; or
2) Childhood Onset: Patients who were growth hormone deficient during childhood as a result of congenital, genetic, acquired, or idiopathic causes. In general, confirmation of the diagnosis of adult GHD in both groups usually requires an appropriate growth hormone stimulation test.
Important Safety Information
Somatropin should not be used for growth promotion in pediatric patients with closed epiphyses or in patients with actie proliferatie or seere non- proliferatie diabetic retinopathy. Norditropin should not be used in patients with known hypersensitiity to somatropin or any of its excipients.
Somatropin should not be used or should be discontinued with any eidence of actie malignancy. Patients with preexisting malignancy should be monitored carefully for any progression or reoccurrence.
Somatropin should not be used to treat patients with acute critical illness due to complications following open heart or abdominal surgery, multiple accidental trauma or acute respiratory failure as increased mortality may occur.
Deaths hae been reported in patients with Prader-Willi syndrome who are seerely obese or hae seere respiratory impairment and are treated with somatropin. Unless patients with Prader-Willi syndrome also hae a diagnosis of GHD, Norditropin is not indicated for the treatment of patients who hae growth failure due to genetically confirmed Prader-Willi syndrome.
Blood glucose leels should be monitored periodically as treatment with somatropin may decrease insulin sensitiity. Patients with preexisting diabetes or glucose intolerance should be monitored closely during somatropin therapy. Doses of insulin or oral agents may need to be adjusted for patients with diabetes on somatropin therapy.
Intracranial hypertension (IH) with papilledema, isual changes, headache, nausea and/or omiting has been reported in a small number of patients treated with somatropin products. Symptoms usually occurred within the first eight weeks after initiation of somatropin therapy and generally resole after cessation of therapy or a reduction of the somatropin dose. Funduscopic examination should be performed routinely before initiating and periodically during the course of somatropin therapy. If papilledema is obsered by funduscopy during somatropin treatment, treatment should be discontinued.
Pediatric patients may deelop slipped capital femoral epiphyses more frequently if they hae endocrine disorders or during rapid growth. Any child haing onset of a limp or complaints of hip or knee pain during somatropin therapy should be carefully ealuated. Progression of scoliosis can occur in patients who experience rapid growth. Somatropin has not been shown to increase the occurrence of scoliosis.
In patients with GHD, central (secondary) hypothyroidism may first become eident or worsen during somatropin treatment. Patients treated with somatropin should therefore hae periodic thyroid function tests and thyroid hormone replacement therapy should be initiated or adjusted as needed.
Somatropin inhibits 11 Beta-hydroxysteroid dehydrogenase type 1 (11 Beta HSD-1) in adipose/hepatic tissue, and may significantly impact the metabolism of cortisol and cortisone. In patients treated with somatropin, preiously undiagnosed central (secondary) hypoadrenalism may be unmasked requiring glucocorticoid replacement therapy. In addition, patients treated with glucocorticoid replacement therapy especially with cortisone acetate and prednisone for preiously diagnosed hypoadrenalism may require an increase in their maintenance or stress doses.
Careful monitoring is adisable when somatropin is administered in combination with other drugs known to be metabolized by CP450 lier enzymes (e.g., corticosteroids, sex steroids, anticonulsants, cyclosporine) or other hormone replacement therapy.
The safety and effectieness of Norditropin in patients age 65 years and older has not been ealuated in clinical studies. Elderly patients may be more sensitie to the actions of somatropin and may be more prone to deelop aderse reactions.
Common somatropin-related aderse reactions include injection site reactions/rashes, lipoatrophy and headaches, glucose intolerance, fluid retention and unmasking of latent central hypothyroidism.
Most serious aderse reactions include intracranial hypertension, diabetic retinopathy, glucose intolerance, slipped capital femoral epiphysis, progression of preexisting scoliosis, sudden death in pediatric patients with Prader-Willi syndrome with risk factors including seere obesity, history of upper airway obstruction or sleep apnea and unidentified respiratory infection, intracranial tumors,
For prescribing information, please isit http://www.norditropin-us.com.
About Noo Nordisk
Noo Nordisk is a healthcare company and a world leader in diabetes care. The company has the broadest diabetes product portfolio in the industry, including the most adanced products within the area of insulin deliery systems. In addition, Noo Nordisk has a leading position within areas such as hemostasis management, growth hormone therapy and hormone replacement therapy. Noo Nordisk manufactures and markets pharmaceutical products and serices that make a significant difference to patients, the medical profession and society. With headquarters in Denmark, Noo Nordisk employs more than 22,750 employees in 79 countries, and markets its products in 179 countries. For more information, please isit http://noonordisk-us.com.
Noo Nordisk
http://noonordisk-us.com
http://www.medicalnewstoday.com/medicalnews.php?newsid=72886
FDA批准Norditropin（R）用于治疗由一种罕见遗传障碍引起的身材矮小症。
Main Category: Endocrinology News
主分类；内分泌学资讯
Article Date: 02 Jun 2007 - 0:00 PDT
文章日期：2007年6月2日美国太平洋白昼时间0：00
Noo Nordisk today announced that Norditropin(R) (somatropin[rDNA origin] injection)receied U.S. Food and Drug Administration (FDA) approal for the treatment of short stature in children with Noonan syndrome. Noonan syndrome is defined as an autosomal dominant genetic syndrome commonly characterized by short stature, congenital heart defects, and unique facial features. The appearance of this disorder can include hypertelorism (widely-set eyes), down-slanting eyes, webbed neck, and other conditions, including congenital heart disease in half of those affected. Up to 80 percent of children with Noonan syndrome suffer from significant short stature.
诺和诺德公司今天宣布Norditropin（R）（rDNA来源的生长激素注射液）获得了美国食品药品监管局（FDA）批准，用于治疗努南综合症儿童的身材矮小症。努南综合症被定义为一种常染色体显性的遗传性综合症，其一般特征为身材矮小、先天性心脏缺损、以及独特的面部特征。这种病症的表象可能包括器官距离过远（双目远离）、向下斜视、蹼状颈、以及其它状况，包括半数患有先天性心脏病。80%努南综合症患儿表现明显的身材矮小。
"When you see a child who has Noonan syndrome, it may not always be obious to the naked eye, but the complications associated with the condition are quite serious and can affect both physical deelopment and other systems" said Martin Soeters, president of Noo Nordisk Inc. "There are few treatment options aailable to help the physical deelopment, and this approal marks an exciting adancement for children with this rare condition."
“当你看到一个患有努南综合症的孩子，在肉眼看上去并不总是那么显眼，但是与此病症相关的并发症却是相当严重的，会影响到身体发育和其它系统。”诺和诺德公司总裁Martin Soeters说，“没有多少治疗方案可以帮助身体发育，而这次批准对于患有此罕见状况的儿童来说，标志着一个激动人心的进步”
Noonan syndrome is classified as a rare condition with a population of less than 200,000. To encourage the deelopment of treatments for rare disorders -- that may not otherwise be commercially iable for deelopment -- the FDA designates drugs that treat fewer than 200,000 patients with an "orphan drug" designation. Norditropin has receied orphan drug designation for the treatment of short stature associated with Noonan syndrome.
努南综合症被划分为一种患病人群少于200000的罕见状况。为了激励对于罕见疾病的治疗开发，FDA将治疗患病人群少于200000的药物指定为“孤儿药”，否则这些开发项目可能没有商业意义。诺和诺德已经获得“孤儿药”指定，以治疗努南综合症引发的儿童身材矮小。
About Noonan Syndrome
关于努南综合症
The prealence of Noonan syndrome has not been determined accurately to date, but most authors report 1 in 1,000 - 2,500 lie births, affecting males and females equally. Based on the United States population, prealence may range anywhere from 125,000 to 300,000 lie births. Howeer, fetal loss can occur in Noonan syndrome so actual incidence of the disorder may be higher than its prealence.
直至今日，努南综合症的患病率尚未准确测得，但大多数作者的报告是在1000到2500名活产儿中即有一例，男女患病机率相等。基于美国人口，各地的患病率从125000到300000名活产儿。然而，努南综合症患儿可能夭折，因此此病的实际发生率将比其患病率高。
Clinical Features and Complications
临床特征及并发症
-- Unique facial features, such as widely-spaced eyes, triangular face, low-set ears, and short-webbed neck
面部特征奇特，例如双目距离远，三角脸，低位耳以及短蹼颈。
-- Short stature (up to 80% of indiiduals)
身材矮小（高达80%个体患有此症）
-- Congenital heart defects
先天性心脏缺损
-- Abnormal chest (shrunken sternum or concae chest)
胸部畸型（缩胸或凹胸）
-- Undescended testes in males (more than 50%)
男性隐睾（超过50%）
-- Bleeding disorders, and easy bruising, especially Factor XI (clotting factor) deficiency (which causes Hemophilia C)
出血障碍，易受创伤，特别是XI因子（凝血因子）缺乏（其导致血友病C）
-- Feeding difficulties with babies, including poor suckling and weaning; frequent or forceful omiting may also occur
婴儿喂养困难，包括哺乳及断乳，频率或剧烈的呕吐也可能发生
-- Common eye problems including near sightedness and a squint
一般的眼病，包括近视和斜视
-- Hearing problems caused by middle ear infections
中耳感染导致的听力问题
-- Poor muscle tone in early deelopment
早期出现的肌张力差
-- Lymphatic system problems, such as lymphedema
淋巴系统疾病，例如淋巴水肿
There is no specific pharmacologic therapy currently aailable, and treatment for Noonan syndrome focuses on its clinical features and complications.
目前尚无特定的药理治疗可用，努南综合症的治疗集中在其临床特征及并发症。
"Noonan syndrome is a heterogeneous genetic condition in which the clinical features are quite ariable. Short stature, which can be seere, is one of the most common characteristics. Treatment with Norditropin may help children with Noonan syndrome improe one of the most concerning physical features of the condition," said Alicia Romano, M.D., Pediatric Endocrinologist, New York Medical College.
“努南综合症是一种异型遗传状态，其临床特征多变。可能会很严重的身材矮小，是其最常见的特征。采用Norditropin治疗能够帮助努南综合症患儿提高一个最关心体态问题。”
纽约医学院儿科内分泌学家Alicia Romano博士说。
About Norditropin
关于Norditropin
Norditropin(R) (somatropin [rDNA origin] injection) is indicated for the treatment of children with short stature associated with Noonan syndrome, treatment of children with growth failure due to inadequate secretion of endogenous growth hormone and for replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD) who meet either of the following two criteria:
Norditropin（R）（rDNA来源的生长激素注射液）用于治疗努南综合症引发的儿童身材矮小，治疗由于内源性生长激素分泌不足导致的儿童生长不良，如果**生长激素缺乏（GHD），且符合以下两个标准之一，则其也用于替代内源性生长激素，
1) Adult Onset: Patients who hae GHD, either alone or associated with multiple hormone deficiencies (hypopituitarism), as a result of pituitary disease, hypothalamic disease, surgery, radiation therapy, or trauma; or
1）**发病：患者患有GHD，由于脑下垂体疾病，下丘脑疾病，放射疗法或创伤导致的涉及一种或多种激素的激素缺乏症（垂体功能减退症），或者
2) Childhood Onset: Patients who were growth hormone deficient during childhood as a result of congenital, genetic, acquired, or idiopathic causes. In general, confirmation of the diagnosis of adult GHD in both groups usually requires an appropriate growth hormone stimulation test.
2）儿童发病：患者患有由于先天性，遗传性，获得性或原发性原因导致的儿童时期生长激素缺乏。一般来说，对于两组中的**GHD的诊断确认通常需要采用一种适宜的生长激素刺激试验。
Important Safety Information
重要的安全信息
Somatropin should not be used for growth promotion in pediatric patients with closed epiphysis or in patients with actie proliferatie or seere non- proliferatie diabetic retinopathy. Norditropin should not be used in patients with known hypersensitiity to somatropin or any of its excipients.
生长激素不应用于儿科闭合骺患者，活性增生或严重非增生糖尿病视网膜病患者的生长促进。Norditropin不应用于已知对生长激素或其中任何辅料过敏的患者。
Somatropin should not be used or should be discontinued with any eidence of actie malignancy. Patients with preexisting malignancy should be monitored carefully for any progression or reoccurrence.
如有证据表明患者患有活性恶性肿瘤，则不应使用生长激素，或应停用。有即成恶性肿瘤的患者应仔细监测其进展或复发。
Somatropin should not be used to treat patients with acute critical illness due to complications following open heart or abdominal surgery, multiple accidental trauma or acute respiratory failure as increased mortality may occur.
生长激素不应用于治疗开胸或腹部外科，多重意外伤或急性呼吸衰竭后因并发症急性病危的病人，因为死亡率会升高。
Deaths hae been reported in patients with Prader-Willi syndrome who are seerely obese or hae seere respiratory impairment and are treated with somatropin. Unless patients with Prader-Willi syndrome also hae a diagnosis of GHD, Norditropin is not indicated for the treatment of patients who hae growth failure due to genetically confirmed Prader-Willi syndrome.
患严重肥胖或严重呼吸损伤的帕-魏二氏综合症，并使用生长激素治疗患者中，有死亡病例的报导。除非患帕-魏二氏综合症的患者也被诊断患有GHD，Norditropin不应用于治疗因遗传学确认的帕-魏二氏综合症导致的生长不良患者。
Blood glucose leels should be monitored periodically as treatment with somatropin may decrease insulin sensitiity. Patients with preexisting diabetes or glucose intolerance should be monitored closely during somatropin therapy. Doses of insulin or oral agents may need to be adjusted for patients with diabetes on somatropin therapy.
生长激素能降低胰岛素的敏感性，因此应周期性监测血糖水平。患糖尿病或葡萄糖耐受不良的患者，在生长激素治疗期间应严密监测。患有糖尿病并进行生长激素治疗的患者，需要调节胰岛素或口服剂的剂量。
Intracranial hypertension (IH) with papilledema, isual changes, headache, nausea and/or omiting has been reported in a small number of patients treated with somatropin products. Symptoms usually occurred within the first eight weeks after initiation of somatropin therapy and generally resole after cessation of therapy or a reduction of the somatropin dose. Funduscopic examination should be performed routinely before initiating and periodically during the course of somatropin therapy. If papilledema is obsered by funduscopy during somatropin treatment, treatment should be discontinued.
伴有视神经乳头水肿，视觉变化，头痛，恶心和/或呕吐的颅内高血压（IH）在使用生长激素产品的少数患者中已经有报导。症状通常在启动生长激素治疗和停止治疗或生长激素剂量量减少后普遍起效的前八周出现。在启动前应进行常规的眼底镜检查，并在生长激素治疗期间周期性检查。在生长激素治疗期间如果通过眼底镜观察到视神经乳头水肿，应停止该治疗。
Pediatric patients may deelop slipped capital femoral epiphyses more frequently if they hae endocrine disorders or during rapid growth. Any child haing onset of a limp or complaints of hip or knee pain during somatropin therapy should be carefully ealuated. Progression of scoliosis can occur in patients who experience rapid growth. Somatropin has not been shown to increase the occurrence of scoliosis.
如果患有内分泌病症或正处于快速增长期，儿科患者会更频繁地出现大股骨骺滑动。在生长激素治疗期间，出现跛行或诉说髋关节、膝关节疼痛的儿童，应谨慎评估。脊柱侧凸的恶化可能在经历快速生长的病人中出现。生长激素并未显示出提高脊柱侧凸的发生率。
In patients with GHD, central (secondary) hypothyroidism may first become eident or worsen during somatropin treatment. Patients treated with somatropin should therefore hae periodic thyroid function tests and thyroid hormone replacement therapy should be initiated or adjusted as needed.
患有GHD的患者，在采用生长激素治疗时，中央（次级）肾上腺机能衰退会首次变得明显或恶化。因此采用生长激素治疗的患者应该进行周期性的甲状腺机能试验，如果需要，应启动或调节甲状腺激素替代治疗。
Somatropin inhibits 11 Beta-hydroxy steroid dehydrogenase type 1 (11 Beta HSD-1) in adipose/hepatic tissue, and may significantly impact the metabolism of cortisol and cortisone. In patients treated with somatropin, preiously undiagnosed central (secondary) hypoadrenalism may be unmasked requiring glucocorticoid replacement therapy. In addition, patients treated with glucocorticoid replacement therapy especially with cortisone acetate and prednisone for preiously diagnosed hypoadrenalism may require an increase in their maintenance or stress doses.
生长激素抑制脂肪和肝组织中的1型11β-羟基类固醇脱氢酶（11 Beta HSD-1），并能明显影响皮质醇和肾上腺皮质激素的代谢。在使用生长激素治疗的病人中，早期不能诊断出中央（次级）肾上腺机能衰退的，会暴露出需要糖皮质激素替代治疗。另外，早期诊断肾上腺机能衰退并采用糖皮质激素替代治疗特别是采用醋酸可的松和泼尼松治疗的患者，会需要提高维持或应激剂量。
Careful monitoring is adisable when somatropin is administered in combination with other drugs known to be metabolized by CP450 lier enzymes (e.g., corticosteroids, sex steroids, anticonulsants, cyclosporine) or other hormone replacement therapy.
当生长激素与其它由CP450肝酶代谢的药物（例如皮质激素，性甾体，抗惊厥剂，环孢菌A）或其它激素替代治疗合用时，建议严密监测。
The safety and effectieness of Norditropin in patients age 65 years and older has not been ealuated in clinical studies. Elderly patients may be more sensitie to the actions of somatropin and may be more prone to deelop aderse reactions.
Norditropin在65岁以上患者中的安全性和有效性尚未在临床研究中评估。老年患者对生长激素的作用会更敏感，而且会更易于出现不良反应。
Common somatropin-related aderse reactions include injection site reactions/rashes, lipoatrophy and headaches, glucose intolerance, fluid retention and unmasking of latent central hypothyroidism.
常见的与生长激素相关的不良反应包括注射部位反应/疹，皮下脂肪萎缩和头痛，葡萄糖耐受不良，液体潴留，潜在的中枢性甲状腺功能减退症的暴露。
Most serious aderse reactions include intracranial hypertension, diabetic retinopathy, glucose intolerance, slipped capital femoral epiphysis, progression of preexisting scoliosis, sudden death in pediatric patients with Prader-Willi syndrome with risk factors including seere obesity, history of upper airway obstruction or sleep apnea and unidentified respiratory infection, intracranial tumors,
最严重的不良反应包括颅内高血压、糖尿病性视网膜病，葡萄糖耐受不良、头骨骺脱位、预成脊柱侧凸的恶化、包括严重肥胖、有上呼吸道梗阻史或睡眠呼吸暂停、不确定性呼吸道感染、颅内肿瘤等高风险因子的帕-魏二氏综合症患儿猝死。
For prescribing information, please isit http://www.norditropin-us.com.
处方信息，请访问：http://www.norditropin-us.com.
About Noo Nordisk
关于诺和诺德
Noo Nordisk is a healthcare company and a world leader in diabetes care. The company has the broadest diabetes product portfolio in the industry, including the most adanced products within the area of insulin deliery systems. In addition, Noo Nordisk has a leading position within areas such as hemostasis management, growth hormone therapy and hormone replacement therapy. Noo Nordisk manufactures and markets pharmaceutical products and serices that make a significant difference to patients, the medical profession and society. With headquarters in Denmark, Noo Nordisk employs more than 22,750 employees in 79 countries, and markets its products in 179 countries. For more information, please isit http://noonordisk-us.com.
诺和诺德是一家医疗保健公司，同时也是糖尿病护理的世界领先者。公司拥有行业内最广泛的糖尿病产品，包括在胰岛素传递系统领域最先进的产品。另外，诺和诺德在止血处理，生长激素治疗和激素替代治疗等领域处于领先地位。诺和诺德为患者，医务和协会生产并销售明显与众不同的药品和服务。诺和诺德总部位于丹麦，在79个国家雇佣超过22750名雇员，其产品在179个国家上市销售。更多信息，请访问http://noonordisk-us.com.
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FDA批准Norditropin（R）用于治疗由一种罕见遗传障碍引起的身材矮小症
主分类；内分泌学资讯
文章日期：2007年6月2日美国太平洋白昼时间0：00
诺和诺德公司今天宣布Norditropin（R）（rDNA来源的生长激素注射液）获得了美国食品药品监管局（FDA）批准，用于治疗努南综合症儿童的身材矮小症。努南综合症被定义为一种常染色体显性的遗传性综合症，其一般特征为身材矮小、先天性心脏缺损、以及独特的面部特征。这种病症的表象可能包括器官距离过远（双目远离）、向下斜视、蹼状颈、以及其它状况，包括半数患有先天性心脏病。80%努南综合症患儿表现明显的身材矮小。
“当你看到一个患有努南综合症的孩子，在肉眼看上去并不总是那么显眼，但是与此病症相关的并发症却是相当严重的，会影响到身体发育和其它系统。”诺和诺德公司总裁Martin Soeters说，“没有多少治疗方案可以帮助身体发育，而这次批准对于患有此罕见状况的儿童来说，标志着一个激动人心的进步”
努南综合症被划分为一种患病人群少于200000的罕见状况。为了激励对于罕见疾病的治疗开发，FDA将治疗患病人群少于200000的药物指定为“孤儿药”，否则这些开发项目可能没有商业意义。诺和诺德已经获得“孤儿药”指定，以治疗努南综合症引发的儿童身材矮小。
关于努南综合症
直至今日，努南综合症的患病率尚未准确测得，但大多数作者的报告是在1000到2500名活产儿中即有一例，男女患病机率相等。基于美国人口，各地的患病率从125000到300000名活产儿。然而，努南综合症患儿可能夭折，因此此病的实际发生率将比其患病率高。
临床特征及并发症面部特征奇特，例如双目距离远，三角脸，低位耳以及短蹼颈。
身材矮小（高达80%个体患有此症）
先天性心脏缺损
胸部畸型（缩胸或凹胸）
男性隐睾（超过50%）
出血障碍，易受创伤，特别是XI因子（凝血因子）缺乏（其导致血友病C）
婴儿喂养困难，包括哺乳及断乳，频率或剧烈的呕吐也可能发生
一般的眼病，包括近视和斜视
中耳感染导致的听力问题
早期出现的肌张力差
淋巴系统疾病，例如淋巴水肿
目前尚无特定的药理治疗可用，努南综合症的治疗集中在其临床特征及并发症。
“努南综合症是一种异型遗传状态，其临床特征多变。可能会很严重的身材矮小，是其最常见的特征。采用Norditropin治疗能够帮助努南综合症患儿提高一个最关心体态问题。”
纽约医学院儿科内分泌学家Alicia Romano博士说。
关于Norditropin
Norditropin（R）（rDNA来源的生长激素注射液）用于治疗努南综合症引发的儿童身材矮小，治疗由于内源性生长激素分泌不足导致的儿童生长不良，如果**生长激素缺乏（GHD），且符合以下两个标准之一，则其也用于替代内源性生长激素，
1）**发病：患者患有GHD，由于脑下垂体疾病，下丘脑疾病，放射疗法或创伤导致的涉及一种或多种激素的激素缺乏症（垂体功能减退症），或者
2）儿童发病：患者患有由于先天性，遗传性，获得性或原发性原因导致的儿童时期生长激素缺乏。一般来说，对于两组中的**GHD的诊断确认通常需要采用一种适宜的生长激素刺激试验。
重要的安全信息
生长激素不应用于儿科闭合骺患者，活性增生或严重非增生糖尿病视网膜病患者的生长促进。Norditropin不应用于已知对生长激素或其中任何辅料过敏的患者。
如有证据表明患者患有活性恶性肿瘤，则不应使用生长激素，或应停用。有即成恶性肿瘤的患者应仔细监测其进展或复发。
生长激素不应用于治疗开胸或腹部外科，多重意外伤或急性呼吸衰竭后因并发症急性病危的病人，因为死亡率会升高。
患严重肥胖或严重呼吸损伤的帕-魏二氏综合症，并使用生长激素治疗患者中，有死亡病例的报导。除非患帕-魏二氏综合症的患者也被诊断患有GHD，Norditropin不应用于治疗因遗传学确认的帕-魏二氏综合症导致的生长不良患者。
生长激素能降低胰岛素的敏感性，因此应周期性监测血糖水平。患糖尿病或葡萄糖耐受不良的患者，在生长激素治疗期间应严密监测。患有糖尿病并进行生长激素治疗的患者，需要调节胰岛素或口服剂的剂量。
伴有视神经乳头水肿，视觉变化，头痛，恶心和/或呕吐的颅内高血压（IH）在使用生长激素产品的少数患者中已经有报导。症状通常在启动生长激素治疗和停止治疗或生长激素剂量量减少后普遍起效的前八周出现。在启动前应进行常规的眼底镜检查，并在生长激素治疗期间周期性检查。在生长激素治疗期间如果通过眼底镜观察到视神经乳头水肿，应停止该治疗。
如果患有内分泌病症或正处于快速增长期，儿科患者会更频繁地出现大股骨骺滑动。在生长激素治疗期间，出现跛行或诉说髋关节、膝关节疼痛的儿童，应谨慎评估。脊柱侧凸的恶化可能在经历快速生长的病人中出现。生长激素并未显示出提高脊柱侧凸的发生率。
患有GHD的患者，在采用生长激素治疗时，中央（次级）肾上腺机能衰退会首次变得明显或恶化。因此采用生长激素治疗的患者应该进行周期性的甲状腺机能试验，如果需要，应启动或调节甲状腺激素替代治疗。
生长激素抑制脂肪和肝组织中的1型11β-羟基类固醇脱氢酶（11 Beta HSD-1），并能明显影响皮质醇和肾上腺皮质激素的代谢。在使用生长激素治疗的病人中，早期不能诊断出中央（次级）肾上腺机能衰退的，会暴露出需要糖皮质激素替代治疗。另外，早期诊断肾上腺机能衰退并采用糖皮质激素替代治疗特别是采用醋酸可的松和泼尼松治疗的患者，会需要提高维持或应激剂量。
当生长激素与其它由CP450肝酶代谢的药物（例如皮质激素，性甾体，抗惊厥剂，环孢菌A）或其它激素替代治疗合用时，建议严密监测。
Norditropin在65岁以上患者中的安全性和有效性尚未在临床研究中评估。老年患者对生长激素的作用会更敏感，而且会更易于出现不良反应。
常见的与生长激素相关的不良反应包括注射部位反应/疹，皮下脂肪萎缩和头痛，葡萄糖耐受不良，液体潴留，潜在的中枢性甲状腺功能减退症的暴露。
最严重的不良反应包括颅内高血压、糖尿病性视网膜病，葡萄糖耐受不良、头骨骺脱位、预成脊柱侧凸的恶化、包括严重肥胖、有上呼吸道梗阻史或睡眠呼吸暂停、不确定性呼吸道感染、颅内肿瘤等高风险因子的帕-魏二氏综合症患儿猝死。
处方信息，请访问：http://www.norditropin-us.com.
关于诺和诺德
诺和诺德是一家医疗保健公司，同时也是糖尿病护理的世界领先者。公司拥有行业内最广泛的糖尿病产品，包括在胰岛素传递系统领域最先进的产品。另外，诺和诺德在止血处理，生长激素治疗和激素替代治疗等领域处于领先地位。诺和诺德为患者，医务和协会生产并销售明显与众不同的药品和服务。诺和诺德总部位于丹麦，在79个国家雇佣超过22750名雇员，其产品在179个国家上市销售。更多信息，请访问http://noonordisk-us.com.
诺和诺德
http://noonordisk-us.com
http://www.medicalnewstoday.com/medicalnews.php?newsid=72886
widely-set eyes 可翻成眼距过宽
unique facial features 常翻成特殊面容
发表者QINQIN 时间 2010-06-21 10:17:55 AM
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